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Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
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Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
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Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
×
Page 7
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
×
Page 8
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
×
Page 9
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
×
Page 10
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
×
Page 11
Suggested Citation:"1 Introduction." National Academies of Sciences, Engineering, and Medicine. 2021. Building Data Capacity for Patient-Centered Outcomes Research: Interim Report Two - Data Standards, Methods, and Policy. Washington, DC: The National Academies Press. doi: 10.17226/26298.
×
Page 12

Below is the uncorrected machine-read text of this chapter, intended to provide our own search engines and external engines with highly rich, chapter-representative searchable text of each book. Because it is UNCORRECTED material, please consider the following text as a useful but insufficient proxy for the authoritative book pages.

  PREPUBLICATION COPY, Uncorrected Proofs    1 Introduction The Office of the Assistant Secretary for Planning and Evaluation (ASPE), in partnership with other agencies and divisions of the U.S. Department of Health and Human Services (HHS), coordinates a portfolio of projects that build data capacity for conducting patient-centered outcomes research (PCOR). The PCOR data infrastructure provides decision makers with objective, scientific evidence on the effectiveness of treatments, services, and other interventions used in health care. This research is frequently focused on analyzing existing data to address questions and provide objective information for the purpose of informing real-world health care decisions. BACKGROUND The legal framework that established funding for research on the outcomes and effectiveness of treatments and health care interventions dates back to the 2003 Medicare Prescription Drug, Improvement, and Modernization Act. This act provided authorization for the Agency for Healthcare Research and Quality (AHRQ) to support research comparing the outcomes and effectiveness of treatments and clinical approaches and to disseminate the findings from this research. In 2009, the American Recovery and Reinvestment Act provided additional funding to AHRQ, the National Institutes of Health, and HHS for research that compares the effectiveness of medical options. In 2010, the Patient Protection and Affordable Care Act provided further authorization for research that assists patients, clinicians, purchasers, and policy makers in making informed health decisions. To facilitate patient-centered outcomes research, in 2010 Congress established the Patient-Centered Outcomes Research Trust Fund (PCOR Trust Fund) with the Department of Treasury. The goals of the PCOR Trust Fund are to fund PCOR research, disseminate research findings, and develop a data infrastructure for PCOR. The PCOR Trust Fund has been reauthorized through 2029, through H.R.1865 of the Further Consolidated Appropriations Act of 2020. The most recent statute specified intellectual and developmental disabilities, as well as maternal mortality, as research priorities. The statute also called for PCOR studies to include consideration of the full range of outcomes data. Specifically, the law states that: Research shall be designed, as appropriate, to take into account and capture the full range of clinical and patient- centered outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers in making informed health decisions. In addition to the relative health outcomes and clinical effectiveness, clinical and patient- centered outcomes shall include the potential burdens and economic impacts of the utilization of medical treatments, items, and services on different stakeholders and decision-makers respectively. These potential burdens and economic impacts include medical out-of-pocket costs, including health plan benefit and formulary design, non-medical costs to the 5  

  PREPUBLICATION COPY, Uncorrected Proofs    patient and family, including caregiving, effects on future costs of care, workplace productivity and absenteeism, and healthcare utilization.2 The bulk of the PCOR Trust Fund funding (80 %) is allocated for research and is made available through the Patient-Centered Outcomes Research Institute (PCORI), a nongovernmental organization established by Congress for this purpose. Approximately 16 percent of the PCOR Trust Fund funding is set aside for disseminating research findings, incorporating findings into clinical practice, and training researchers in patient-centered outcomes research. The agency overseeing this work is AHRQ. The remaining funding, which constitutes 4 percent of the PCOR Trust Fund, is allocated for building data capacity for PCOR and is overseen by ASPE. Specifically, Section 937(f) of the Public Health Service Act instructed the Secretary of HHS to: … provide for the coordination of relevant Federal health programs to build data capacity for comparative clinical effectiveness research, including the development and use of clinical registries and health outcomes research networks, in order to develop and maintain a comprehensive, interoperable data network to collect, link, and analyze data on outcomes and effectiveness from multiple sources including electronic health records.3 Figure 1-1 shows how the PCOR funding and work is allocated across the three entities. This National Academies study is focused on issues relevant to ASPE’s continued work on the PCOR data infrastructure, in other words, on the priorities for the use of the 4 percent of the funding that is allocated to HHS for work related to the data infrastructure for PCOR. As the coordinating agency for the data infrastructure investment portfolio across HHS agencies, ASPE guides the PCOR data infrastructure’s strategic framework and vision, sets funding priorities, and coordinates interagency workgroups. ASPE’s work is assisted by a Leadership Council for the PCOR Trust Fund, which includes representatives from other HHS agencies, including the Administration for Children and Families, the Administration for Community Living, the Assistant Secretary for Preparedness and Response, AHRQ, the Centers for Disease Control and Prevention (CDC), the Centers for Medicare & Medicaid Services, the Food and Drug Administration (FDA), the Health Resources and Services Administration, the Indian Health Service, the National Institutes of Health, the Office of the Chief Technology Officer, the Office of the National Coordinator for Health Information Technology, and the Substance Abuse and Mental Health Services Administration. The Leadership Council provides input on priorities for the portfolio, including projects to fund. During the period from 2010 to 2019, the PCOR Trust Fund funded 53 projects, which translated to 76 agency awards, totaling approximately $131 million.                                                              2 https://www.ssa.gov/OP_Home/ssact/title11/1181.htm. 3 https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/about-os-pcortf. 6  

  PREPUBLICATION COPY, Uncorrected Proofs    FIGURE 1-1 Patient-Centered Outcomes Research Trust Fund: three streams of work and funding. NOTE: AHRQ = Agency for Healthcare Research and Quality; DHHS = Department of Health and Human Services; PCOR = patient-centered outcomes research; PCORI = Patient-Centered Outcomes Research Institute. SOURCE: Workshop presentation by ASPE, May 3, 2021. Figure 1-2 is a visual representation of ASPE’s current framework for the patient- centered outcomes research data infrastructure. The bottom row shows the main data sources feeding into the PCOR infrastructure. Data collected as part of clinical care include data collected for health care delivery and for billing purposes. Examples of primary data collected as part of research studies include data from clinical trials and national health surveys. Other examples of data sources include: Medicare or Medicaid claims data; quality or outcomes data collected by health care providers for the purposes of improving health care value; FDA data on the safety of medications and medical devices; and CDC data on births and deaths provided by state public health authorities. The framework describes the relationship between the data sources and the current key functionalities and focus areas (middle row) which support the research. The key functionalities are described in further detail in Box 1-1. Major building blocks are the services, standards, policies, and governance that enable the use of the data for research, described in further detail in Box 1-2. The top row shows the key data users and contributors of data. A more detailed overview of ASPE’s work and the projects funded to date will be included in the final report, at the conclusion of the committee’s review. 7  

  PREPUBLICATION COPY, Uncorrected Proofs    FIGURE 1-2 ASPE’s strategic framework for the patient-centered outcomes research data infrastructure. SOURCE: Workshop presentation by ASPE, May 3, 2021. BOX 1-1 Key Data Infrastructure Functionalities in the Existing Strategic Framework for Patient-Centered Outcomes Research Standardized Collection of Standardized Clinical Data Researchers will be able to use standardized clinical data based on common data element standards across research projects and networks, thereby facilitating linkage and aggregation of data across data sources. Collection of Participant-Provided Information Participants, including those in safety net organizations, will be able to participate more fully in clinical research by directly providing information (i.e., data points provided by the participant such as Patient-Reported Outcomes). Linking of Clinical and Other Data for Research Researchers will be able to follow patients across the care continuum over time, including those enrolled in clinical trials. Researchers will be able to capture the range of variables influencing health outcomes and link clinical and other types of data (e.g., other clinical data, claims data, participant-provided information, and environmental data) required for research regardless of where the participant goes. 8  

  PREPUBLICATION COPY, Uncorrected Proofs    Use of Clinical Data for Research Researchers will be able to utilize and analyze routinely collected clinical data for implementation of clinical studies (observational and interventional), including data relevant to assessing safety, efficacy, and adherence, as well as genetic data and Patient-Reported Outcomes. Use of Enhanced Publicly Funded Data Systems for Research Researchers will be able to readily use, retrieve, link, and aggregate publicly funded data for research due to enhancements in publicly funded data systems. SOURCE: https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/about- os-pcortf/building-data-capacity-patient-centered-outcomes-research. BOX 1-2 Building Blocks of the Patient-Centered Outcomes Research Data Infrastructure Standards represent information and meaning to patient-centered data to ensure that health specific information can be accurately (and securely) exchanged and used. In most cases standards should be nationally accepted, widely approved, or broadly adopted either through market forces, community approval or regulatory requirements. These include such items as data standards for capturing, storing, representing, and exchanging data in a secure manner such that accurate information is conveyed to the recipient of the data. Policies are standards of behavior that participants can rely on consistently to build patient- centered data for research. Policies may include federal policies, as well as models for standardized state and local policies, that will lead to a trusted framework within the patient- centered outcomes research (PCOR) data infrastructure that ensures productivity, protects the patient and the patient’s data, ensures that evidence generations remains in the center of PCOR, and ensures the use of agreed upon standards and services. Services refer to resources that entities can employ on demand to capture, store, or exchange either PCOR data or evidence through a centrally hosted model provided remotely (such as through the internet) rather that provided locally or on-site. Services make it easy for the research data to interoperate among different systems without having to start from scratch for every connection. Governance structures refer to entities that are needed to develop and apply the rules and policies needed for building an interoperable and sustainable research network. Governance structures support the efficient use of the data infrastructure for research across individual and organizations’ boundaries of control and ownership. Governance structures are distinguished from “governance” which is what a governing body or governance structure does. 9  

  PREPUBLICATION COPY, Uncorrected Proofs    SOURCE: https://aspe.hhs.gov/collaborations-committees-advisory-groups/os-pcortf/about- os-pcortf/building-data-capacity-patient-centered-outcomes-research. ISSUES FOR THE COMMITTEE ASPE asked the National Academies of Sciences, Engineering, and Medicine to appoint a consensus study committee and identify issues critical to building data capacity for PCOR and for generating new evidence to inform health care decisions. The input provided by the committee will contribute to ASPE’s strategic planning for their work related to the data infrastructure over the next decade. The study is part of a broader initiative by ASPE intended to update the strategic plan in light of the reauthorization of the PCOR Trust Fund and advances in health information technology and interoperability tools in recent years. The study is a collaboration of three units of the National Academies: the Committee on National Statistics, the Board on Health Care Services, and the Computer Science and Telecommunications Board. The consensus study committee has a diverse membership, its 15 members including experts with decades of experience, as well as emerging leaders, in the broad fields of: (1) patient-centered outcomes research; (2) research methods, statistics, and demography; (3) computer science and data infrastructure; and (4) patient engagement and patient perspectives. Appendix A contains the biographical sketches of the committee members. As part of its information gathering activities, the committee was asked to organize three workshops to collect input from stakeholders on aspects of the charge developed in consultation with ASPE. The workshops focused on key topics that the committee believed would particularly benefit from broad input from a variety of data users and other stakeholders. The conclusions from each workshop are summarized in a series of interim reports, of which the first centered on emerging data needs. This first interim report summarizes the discussion and conclusions from the first workshop, which focused on looking ahead at data user needs over the next decade. The second workshop in the series centered on data standards, methods, and policies that could make the PCOR data infrastructure more useful. The third workshop discussed research and data collaborations. This report summarizes the discussion and conclusions from the second workshop, which focused on data standards, methods, and policies that could make the PCOR data infrastructure more useful. The third report will discuss research and data collaborations. As an interim report focused on one in a series of information-gathering activities, the scope of this report is limited to a subset of the topics relevant to the committee’s charge and the conclusions reached by the committee are, at this stage, fairly high level. Some aspects of the topics discussed are examined in further detail in other workshops. After completing all of its information gathering activities, the committee will issue a final report, which will integrate and examine these topics in further detail. Box 1-1 shows the committee’s Statement of Task for the overall study. The committee will address this charge in its final report, integrating what was learned from the workshops and from all other forms of input, including public meetings with HHS staff and background documentation available on the history and operations of the PCOR Trust Fund. The final report will contain overall findings and conclusions from the study, on the basis of the committee’s further deliberations and integrated judgment on the input received and materials reviewed. 10  

  PREPUBLICATION COPY, Uncorrected Proofs    BOX 1-3 Statement of Task for the Overall Study The National Academies will appoint an ad hoc committee to conduct a series of three one-day public workshops and develop conclusions to help guide the data capacity development for patient-centered research from 2021 through 2030. Each workshop will seek input from key stakeholders on topics relevant to the committee charge, and the specific focus of each workshop will be determined by the committee in consultation with ASPE. As part of its activities, the committee will also  Consider the published review of the history and trajectory of the Office of the Secretary Patient-Centered Outcomes Research Trust Fund (OS-PCORTF) portfolio of investments and the OS-PCORTF roadmap;  Assess anticipated changes to health care priorities and priorities for health data and their impact on building data capacity into the foreseeable future, as identified by ASPE;  Evaluate the feasibility and utility of developing a phased-in approach to building the interoperable data capacity for patient-centered outcomes research with existing databases in HHS, other Federal Departments and the private sector in a phased approach, such as projects identified in the Cures Act Title III Section 4003 (Interoperability);  Consider other existing legislation, regulations, and the like, as deemed relevant; and  Receive input from individuals or groups that represent stakeholders, including patients and their caregivers or families and their health care providers. The committee will issue interim reports after each public workshop with conclusions, and will produce a final written report with findings and conclusions to help guide a future course to continue building the data capacity for patient-centered research. All reports will follow institutional guidelines and be subject to the National Academies review procedures prior to release. Appendix B shows the agenda for the workshop, which was held on May 24, 2021. The committee’s goal for this event was to bring together researchers and policy experts to - Identify data standards and methods that can make the PCOR data infrastructure more useful for research and other data needs; - Identify data policies that are needed to facilitate the continued development and operation of the PCOR data infrastructure; - Discuss what HHS is best positioned to address and support, and how the agency could maximize resources available for the PCOR data infrastructure (representing 4% of the PCOR trust fund), in the context of the HHS public mission, authorities, programs, and data resources. Invited speakers in each of the sessions were asked to reflect on the general topics above. The specific questions for each session are described in Chapters 2 through 4. An obvious 11  

  PREPUBLICATION COPY, Uncorrected Proofs    limitation of an activity of this type is that only a small number of stakeholders can be invited to speak. To compensate for this limitation, the invited participants included diverse experts working in a variety of areas and on a range of types of projects, including both early career researchers and experts with decades of experience. A recording of the workshop as well as the presentation slides used by the speakers are available on the National Academies website at www.nationalacademies.org/PCORData. Prior to the workshop, information about the event was disseminated through National Academies mailing lists and on the project website. To collect additional stakeholder input, members of the public were invited to provide comments on topics related to the workshop (or any other topic related to the committee’s charge), using a public input form available on the National Academies website. OVERVIEW OF THE REPORT This report is organized around the three main sessions of the workshop: Chapter 2 discuses data standards; Chapter 3 is centered on research methods; and Chapter 4 describes discussions focused on data policies and related infrastructure considerations. The points conveyed by the workshop participants do not necessarily reflect the views of the committee. In each chapter, a summary of the input received is followed by the committee’s conclusions. The conclusions are based primarily on the input collected as part of the workshop, background documentation received from ASPE and other public sources, and the committee members’ synthesis and expert judgment. Because this is an interim report, the committee’s conclusions at this stage are big-picture conclusions, which will be integrated with additional input over the course of the study. 12  

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The Office of the Assistant Secretary for Planning and Evaluation (ASPE), in partnership with other agencies and divisions of the United States Department of Health and Human Services, coordinates a portfolio of projects that build data capacity for conducting patient-centered outcomes research (PCOR). PCOR focuses on producing scientific evidence on the effectiveness of prevention and treatment options to inform the health care decisions of patients, families, and health care providers, taking into consideration the preferences, values, and questions patients face when making health care choices.

ASPE asked the National Academies to appoint a consensus study committee to identify issues critical to the continued development of the data infrastructure for PCOR. The committee's work will contribute to ASPE's development of a strategic plan that will guide their work related to PCOR data capacity over the next decade.

As part of its information gathering activities, the committee organized three workshops to collect input from stakeholders on the PCOR data infrastructure. This report, the second in a series of three interim reports, summarizes the discussion and committee conclusions from the second workshop, focused on data standards, methods, and policies that could make the PCOR data infrastructure more useful in the years ahead. Participants in the workshop included researchers and policy experts working in these areas.

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